Reduced-intensity conditioning is effective for hematopoietic stem cell transplantation in young pediatric patients with Diamond?Blackfan anemia
Reduced-intensity conditioning is effective for hematopoietic stem cell transplantation in young pediatric patients with Diamond?Blackfan anemia
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降低强度预处理对年轻的戴蒙德·布莱克凡贫血儿科患者的造血干细胞移植有效
DOI：
10.1038/s41409-020-01056-1
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发表时间：
2020
影响因子：
4.8
通讯作者：
Ogawa Seishi et al.
中科院分区：
文献类型：
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作者：
Koyamaishi Shun;Ogawa Seishi et al.
异基因造血干细胞移植（HSCT）是治疗血液学表现的Diamond-Blackfan贫血（DBA）的唯一治疗方法。然而，有关DBA患者最佳预处理方案的数据有限。我们回顾性比较了DBA患者接受HSCT时采用清髓性预处理（MAC）或低强度预处理（RIC）方案的结局。这些患者属于2000年至2018年期间在我们医院接受治疗的队列。165例患者中有27例（16.4%）进行了HSCT。HSCT时的中位年龄为3.6岁。干细胞来源包括25例患者的骨髓（HLA匹配的同胞供体，n= 5; HLA不匹配的相关供体，n= 2; HLA匹配/不匹配的无关供体，n= 18）或2例患者的脐带血。MAC或RIC方案分别用于12例和15例患者。所有27例接受HSCT的患者均成功植入。3例采用MAC方案行HSCT的患者发生了窦阻塞综合征。术后3年总生存率（OS）和无失败生存率（FFS）分别为95.2%和88.4%，MAC和RIC方案之间无显著差异。我们的数据表明，HSCT使用RIC方案是有效的，并获得良好的OS和FFS的年轻DBA患者的植入。
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy for the hematologic manifestations of Diamond–Blackfan anemia (DBA). However, data regarding the optimal conditioning regimen for DBA patients are limited. We retrospectively compared the outcomes of DBA patients who underwent HSCT using either myeloablative conditioning (MAC) or reduced-intensity conditioning (RIC) regimens. The patients belonged to a cohort treated at our hospitals between 2000 and 2018. HSCT was performed in 27 of 165 patients (16.4%). The median age at the time of HSCT was 3.6 years. Stem cell sources included bone marrow for 25 patients (HLA-matched sibling donors,n= 5; HLA-mismatched related donors,n= 2; HLA-matched/mismatched unrelated donors,n= 18) or cord blood for 2 patients. MAC or RIC regimens were used in 12 and 15 patients, respectively. Engraftment was successful in all 27 patients who underwent HSCT. Three patients who underwent HSCT using MAC regimens developed sinusoidal obstruction syndrome. The 3-year overall survival (OS) and failure-free survival rates (FFS) post-transplantations were 95.2% and 88.4%, respectively, with no significant differences between MAC and RIC regimens. Our data suggest that HSCTs using RIC regimens are effective and obtain engraftment with excellent OS and FFS for young DBA patients.